发明名称 |
TARGETED THERAPEUTIC LYSOSOMAL ENZYME FUSION PROTEINS AND USES THEREOF |
摘要 |
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. |
申请公布号 |
WO2014085621(A1) |
申请公布日期 |
2014.06.05 |
申请号 |
WO2013US72287 |
申请日期 |
2013.11.27 |
申请人 |
BIOMARIN PHARMACEUTICAL INC. |
发明人 |
AOYAGI-SCHARBER, MIKA;CHRISTIANSON, TERESA, MARGARET;DVORAK-EWELL, MELITA;WENDT, DANIEL, J.;LONG, SHINONG;LEBOWITZ, JONATHAN;GOLD, DANIEL, SOLOMON |
分类号 |
C07K14/65;A61K38/30;C07K7/08;C12N1/00;C12N15/62 |
主分类号 |
C07K14/65 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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